BridgeBio’s lands on late-stage limb-girdle muscular dystrophy data

Announcement

The bridgeBio Pharma’s in-progress investigational oral small molecule BBP-418, an under-development study to cure a particular form of limb-girdle muscular dystrophy, has entered a late-stage trial. Following this, the new drug application is planned to proceed by the first half of 2026. The analysts at Mizuho Securities state that the trial results have opened the door to the FDA. The analyst added, “The collaboration with the FDA is predicted either between late 2025 or early 2026. We will get into the argument with the confidence of the interim data; there has to be space for discussing an ability-proven approval path versus accelerated approval.”

The Jefferies analyst added, “The data have shown a ‘best-case’ scenario. The BBP-418 complements favourably in terms of comparison with the SRP-9003. The SRP-9003 is a Sarepta Therapeutics investigational gene therapy for limb-girdle muscular dystrophy (LGMD). We feel content in an AA (accelerated approval).” The positive feedback on the data indicates a successful journey for the data to enter confidently into the next procedures to amalgamate and harness the new drug application that BridgeBio is planning for.

Positive results of the data sound beneficial

BridgeBio’s phase 3 FORTIFY study is at the interim first-ever endpoint at three months, close to doubling αDG, a biomarker that shows muscle balance in LGMD type 2I/R9 patients. The trial also aimed at its secondary endpoints, involving an 82% mitigation in serum creatine kinase. The serum creatine kinase is a marker of muscle damage. Observing the 12 months, the patients have seen progress in pulmonary and ambulatory function.

The patients were able to walk at 0.14 meters/second fastest speed in the 100 meters, in comparison to the previous therapy that covered a fastest speed of 0.27 meters/second. It is considered quicker than those in the placebo arm. The trial participants observed 5% increase difference between placebo and predicted pulmonary volume. The benefits noticed in 12 months are a ‘rare feat for muscle disorders’ according to Jefferies analysts.

The positive results have inaugurated the new space and perspective after news of a patient’s death in a clinical trial for another Sarepta LGMD candidate, SRP-9004. After this, the FDA halted all Sarepta’s LGMD clinical trials. Even though Sarepta is distant from gene therapy, but is still studying the SRP-9003’s development. These efforts will be more encouraged by seeing BridgeBio’s leading data and plans.